Vertex makes $420M preclinical play for DMD gene editing field

Vertex Pharma
Vertex plans to open a genetic therapy research site in the Boston area. (Vertex Pharma)

Vertex Pharmaceuticals is paying $420 million upfront to go after the muscular dystrophy market. The outlay will give Vertex ownership of Exonics and expand its deal with CRISPR Therapeutics, setting it up to use gene editing to treat Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). 

Over the past two years, Vertex has moved deeper and deeper into gene editing through deals such as a co-development pact with CRISPR Therapeutics and a licensing agreement with Merck KGaA. The deals have given the Boston-based company both a pipeline of gene editing programs and a selection of tools it can use to discover and advance additional assets.

Now, Vertex is further building out its capabilities, striking deals and hiring ex-Audentes Therapeutics Chief Scientific Officer John Gray to lead a new genetic therapies research site. The flurry of activity moves Vertex into turf staked out by companies including Pfizer, Sarepta Therapeutics and Solid Biosciences.

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The biggest upfront is tied to the acquisition of Exonics. Vertex is paying $245 million to acquire the biotech, which has used technology licensed from Eric Olson’s lab at the University of Texas Southwestern Medical Center to develop an asset that genetically repaired and restored dystrophin in preclinical models. Vertex could pay out around $750 million more in milestones as the DMD and DM1 programs advance.

In the CRISPR deal, Vertex is paying $175 million upfront to form an exclusive agreement to discover and develop DMD and DM1 gene therapies. The deal gives Vertex rights to CRISPR/Cas9 technology, novel endonucleases, AAV vectors and other current and future CRISPR technologies for use in DMD and DM1 gene editing products.

Vertex will use the resources to develop DMD products independently. In DM1, CRISPR will perform guide RNA research—splitting the costs with Vertex—and choose at the IND filing stage whether to co-develop the drug or receive milestones. All other costs will fall squarely on Vertex, which is on the hook for about $800 million in milestones.

The two deals build out Vertex’s capabilities. 

“We are bringing together the intellectual property, technologies and scientific expertise needed to establish a leading gene editing platform for DMD and DM1,” Vertex CEO Jeffrey Leiden said. “We are continuing to build a toolbox of small molecule and nucleic acid technologies and capabilities.”

Vertex has given Gray a leading role in the use of that toolbox. Gray, who left Audentes at the end of last month, is joining Vertex as senior vice president for genetic therapies in 10 days. The appointment gives Vertex a leader with extensive experience of vectors and other aspects of genetic therapies from stints at Audentes and St. Jude Children's Research Hospital.

Gray arrives at an expanding genetic therapy R&D operation. To accommodate the growth, Vertex plans to open a genetic therapy research site in the Boston area. The site will perform research, vector development and clinical manufacturing for assets including the DMD and DM1 programs.

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